FDA Grants RMAT Status to Bionic Sight's BS01 Gene Therapy

On February 18, 2025, Bionic Sight, Inc., a pioneering biotechnology firm specializing in gene therapy for retinal degenerative diseases, announced a significant milestone. The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for its experimental product, BS01. This designation is aimed at treating patients with retinitis pigmentosa (RP) who are experiencing severe vision loss.

The RMAT designation, part of the 21st Century Cures Act, is designed to expedite the development of cutting-edge regenerative medicine therapies, which include gene therapies for serious health conditions. RMAT offers benefits similar to those of fast track and breakthrough therapy designations, such as early interactions with the FDA, specifically focused on regenerative medicine therapies. With this designation, Bionic Sight is gearing up for its Phase 3 program as it moves closer to seeking marketing approval.

Calvin Roberts, MD, President and CEO of the Lighthouse Guild, an international organization supporting the visually impaired, commented on the RMAT designation, stating that it is a substantial validation of Bionic Sight’s BS01 therapy. He emphasized the rigorous FDA review process that focuses intensely on both safety and efficacy. He also highlighted the therapy's potential not only for people with RP but possibly for other retinal degenerative conditions as well.

The RMAT designation for BS01 was awarded based on early data from Bionic Sight’s ongoing Phase 1/2 clinical trial. Participants in this study who received lower doses of the therapy exhibited modest but consistent improvements in their vision. All five participants showed at least a 100-fold increase in light sensitivity, with some gaining the ability to detect motion. Participants administered the highest doses demonstrated even more significant improvements. Ten out of eleven individuals experienced enhanced visual acuity (BCVA), with seven showing measurable improvements on the EDTRS eye chart, averaging nearly five lines of improvement.

In addition to the improvements in BCVA, those at the "count fingers" vision level gained the ability to recognize shapes, objects, and sometimes colors. Patients with "tunnel vision" noted an increase in their visual field size by threefold and showed marked improvements in maze navigation, evident in reduced completion times and fewer errors. The remaining three high-dose participants, initially at the "light perception" level, improved to "hand motion." Importantly, BS01 demonstrated a favorable safety profile, with no serious product-related adverse events reported. Any adverse events were mild and generally symptom-free.

Bionic Sight's ongoing Phase 1/2 trial, identified as NCT04278131, is an open-label, dose-escalation study focused on patients with advanced-stage RP. The primary objective is to evaluate the safety of BS01, particularly regarding ocular or systemic treatment-related adverse events. Efficacy assessments involve measuring light detection, BCVA, shape and object recognition, and maze navigation skills.

Founded by Dr. Sheila Nirenberg, a professor at the Weill Medical College of Cornell University, Bionic Sight, Inc. is dedicated to developing gene therapy vectors and devices for retinal degenerative diseases. Dr. Nirenberg's groundbreaking work in decoding the retina's neural code to restore vision has earned her prestigious recognition, including a MacArthur "genius" award.